Post-Transcriptional Regulation in Skeletal Muscle Development, Repair, and Disease

Skeletal muscle formation is a complex process that requires tight spatiotemporal control of key myogenic factors. Emerging evidence suggests that RNA processing is crucial for the regulation of these factors, and that multiple post-transcriptional regulatory pathways work dependently and independently of one another to enable precise control of transcripts throughout muscle development and repair. Moreover, disruption of these pathways is implicated in neuromuscular disease, and the recent development of RNA-mediated therapies shows enormous promise in the treatment of these disorders. We discuss the overlapping post-transcriptional regulatory pathways that mediate muscle development, how these pathways are disrupted in neuromuscular disorders, and advances in RNA-mediated therapies that present a novel approach to the treatment of these diseases. RNA processing is crucial for muscle development and repair.Myogenic transcripts are regulated by overlapping post-transcriptional processes.Disruption of one or more of these processes contributes to neuromuscular disease.Recent breakthroughs in RNA-mediated therapies show promise in the treatment of spinal muscular atrophy and amyotrophic lateral sclerosis.Leveraging other post-transcriptional regulatory pathways may reveal alternative therapeutic approaches for other neuromuscular disorders.